Trial Outcomes & Findings for Gene Therapy for Painful Diabetic Neuropathy (NCT NCT01002235)
NCT ID: NCT01002235
Last Updated: 2025-10-06
Results Overview
Treatment-emergent adverse events are any adverse events that occurred after the first dose of Engensis (VM202). This is a dose escalation study. Cohorts of increasing dose will be enrolled sequentially. Dose escalation decisions (permission to treat at higher doses) will be made by the Data Safety Monitoring Board based on review of adverse events and on the occurrence of dose limiting toxicities in each cohort. The decision to proceed to the next higher dose cohort will be made according to the scheme described in the protocol.
COMPLETED
PHASE1/PHASE2
12 participants
Day 0 to Day 365
2025-10-06
Participant Flow
Participant milestones
| Measure |
Engensis 4 mg
Subjects received Intramuscular injections of Engensis to the calf on Days 0 and 14
|
Engensis 8 mg
Subjects received Intramuscular injections to the calf on Days 0 and 14
|
Engensis 16 mg
Subjects received Intramuscular injections of Engensis on Days 0 and 14
|
|---|---|---|---|
|
Overall Study
STARTED
|
4
|
4
|
4
|
|
Overall Study
COMPLETED
|
4
|
4
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Gene Therapy for Painful Diabetic Neuropathy
Baseline characteristics by cohort
| Measure |
Engensis 4 mg
n=4 Participants
Subjects received Intramuscular injections of Engensis to the calf on Days 0 and 14
|
Engensis 8 mg
n=4 Participants
Subjects received Intramuscular injections to the calf on Days 0 and 14
|
Engensis 16 mg
n=4 Participants
Subjects received Intramuscular injections of Engensis on Days 0 and 14
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
60.3 years
STANDARD_DEVIATION 7.0 • n=5 Participants
|
61.3 years
STANDARD_DEVIATION 7.1 • n=7 Participants
|
54.8 years
STANDARD_DEVIATION 12.4 • n=5 Participants
|
58.8 years
STANDARD_DEVIATION 8.8 • n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Day 0 to Day 365Population: The Safety Population included all participants who received at least one dose of Engensis (VM202) from Day 0 to Day 365
Treatment-emergent adverse events are any adverse events that occurred after the first dose of Engensis (VM202). This is a dose escalation study. Cohorts of increasing dose will be enrolled sequentially. Dose escalation decisions (permission to treat at higher doses) will be made by the Data Safety Monitoring Board based on review of adverse events and on the occurrence of dose limiting toxicities in each cohort. The decision to proceed to the next higher dose cohort will be made according to the scheme described in the protocol.
Outcome measures
| Measure |
Engensis 4 mg
n=4 Participants
Subjects received Intramuscular injections of Engensis (VM202) to the calf on Days 0 and 14
|
Engensis 8 mg
n=4 Participants
Subjects received Intramuscular injections of Engensis (VM202) to the calf on Days 0 and 14
|
Engensis 16 mg
n=4 Participants
Subjects received Intramuscular injections of Engensis (VM202) on Days 0 and 14
|
|---|---|---|---|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Subjects with any TEAEs
|
3 Participants
|
2 Participants
|
2 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Injection site reaction
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Foot fracture
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Diarrhoea
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Back pain
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Musculoskeletal pain
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Dry eye
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Retinal vascular disorder
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Dry mouth
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Dyspepsia
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Sinusitis
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Viral infection
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Benign prostatic hyperplasia
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg
Ingrowing nail
|
1 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Days 0, 90, 180, and 365Population: Change from Baseline (Day 0) to visits Day 90, Day 180, and Day 365, in the Intent-to-treat (ITT) population participants who received all assigned doses of Engensis and had evaluable data at a follow-up visit
Percent change in median Visual Analog Scale Pain Scores from baseline (Day 0). The Visual Analog Scale Pain scoring instrument is a 10-cm line, oriented horizontally, with the left end (0 cm, is 0 Percent) indicating "no pain" and the right end (10 cm, is 100 percent) representing "pain as bad as it can be". The Change from Baseline of the Visual Analog Scale Pain Score is the difference between the Day 0 and the Days 90, 180 and 365 scores. A negative difference in the Change from Baseline of the Visual Analog Scale Pain Scores, indicates an improvement (reduction of pain severity) of the treated groups Visual Analog Score.
Outcome measures
| Measure |
Engensis 4 mg
n=4 Participants
Subjects received Intramuscular injections of Engensis (VM202) to the calf on Days 0 and 14
|
Engensis 8 mg
n=4 Participants
Subjects received Intramuscular injections of Engensis (VM202) to the calf on Days 0 and 14
|
Engensis 16 mg
n=4 Participants
Subjects received Intramuscular injections of Engensis (VM202) on Days 0 and 14
|
|---|---|---|---|
|
Percent Change From Baseline in Visual Analog Scale Pain Scores
Day 90
|
-19.19 percentage of Change from Baseline
Standard Deviation 71.53
|
-40.76 percentage of Change from Baseline
Standard Deviation 28.55
|
-40.15 percentage of Change from Baseline
Standard Deviation 21.50
|
|
Percent Change From Baseline in Visual Analog Scale Pain Scores
Day 180
|
-26.16 percentage of Change from Baseline
Standard Deviation 84.35
|
-58.40 percentage of Change from Baseline
Standard Deviation 39.36
|
-57.07 percentage of Change from Baseline
Standard Deviation 30.26
|
|
Percent Change From Baseline in Visual Analog Scale Pain Scores
Day 365
|
-42.27 percentage of Change from Baseline
Standard Deviation 108.26
|
-67.57 percentage of Change from Baseline
Standard Deviation 47.56
|
-46.97 percentage of Change from Baseline
Standard Deviation 29.01
|
Adverse Events
Engensis 4 mg
Engensis 8 mg
Engensis 16 mg
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Engensis 4 mg
n=4 participants at risk
Subjects received Intramuscular injections of Engensis (VM202) to the calf on Days 0 and 14
|
Engensis 8 mg
n=4 participants at risk
Subjects received Intramuscular injections of Engensis (VM202) to the calf on Days 0 and 14
|
Engensis 16 mg
n=4 participants at risk
Subjects received Intramuscular injections of Engensis (VM202) on Days 0 and 14
|
|---|---|---|---|
|
Musculoskeletal and connective tissue disorders
Back pain
|
25.0%
1/4 • Number of events 1 • 365 days
|
25.0%
1/4 • Number of events 1 • 365 days
|
0.00%
0/4 • 365 days
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
25.0%
1/4 • Number of events 1 • 365 days
|
|
Eye disorders
Dry eye
|
25.0%
1/4 • Number of events 2 • 365 days
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
|
Eye disorders
Retinal vascular disorder
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
25.0%
1/4 • Number of events 1 • 365 days
|
|
Gastrointestinal disorders
Diarrhoea
|
25.0%
1/4 • Number of events 1 • 365 days
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
|
Gastrointestinal disorders
Dry mouth
|
25.0%
1/4 • Number of events 1 • 365 days
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
|
Gastrointestinal disorders
Dyspepsia
|
25.0%
1/4 • Number of events 1 • 365 days
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
|
Infections and infestations
Sinusitis
|
0.00%
0/4 • 365 days
|
25.0%
1/4 • Number of events 1 • 365 days
|
0.00%
0/4 • 365 days
|
|
Infections and infestations
Viral infection
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
25.0%
1/4 • Number of events 1 • 365 days
|
|
Infections and infestations
Injection site reaction
|
25.0%
1/4 • Number of events 1 • 365 days
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
|
Injury, poisoning and procedural complications
Foot fracture
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
25.0%
1/4 • Number of events 1 • 365 days
|
|
Reproductive system and breast disorders
Benign prostatic hyperplasia
|
0.00%
0/4 • 365 days
|
25.0%
1/4 • Number of events 1 • 365 days
|
0.00%
0/4 • 365 days
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
25.0%
1/4 • Number of events 1 • 365 days
|
0.00%
0/4 • 365 days
|
0.00%
0/4 • 365 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place